RecruitingOBSERVATIONAL

Natural History of Duchenne Muscular Dystrophy

This research study aims to understand how Duchenne muscular dystrophy (DMD) naturally progresses in boys aged 4 to 9 years old. It's not testing a new treatment, but rather observing the disease over 6 to 36 months to collect important information. The main goals are to track changes in the disease using standard assessments, and to create a baseline understanding before boys might join future gene therapy trials. The study also hopes to find early signs or measurements that can predict how the disease will develop and identify the best ways to measure progress in future clinical trials. This information is crucial for developing better treatments for DMD.

At a glance

Status

Recruiting

Sponsor

Genethon

Enrolment target

220

Start

19 Dec 2019

Estimated completion

30 Sep 2029

Duchenne Muscular Dystrophy

What is this study about?

This study is designed to help doctors and scientists understand Duchenne Muscular Dystrophy (DMD) better. DMD is a condition that causes muscles to weaken over time. This particular study isn't testing a new medicine or treatment; instead, it's about carefully observing how the condition progresses naturally in young boys. Think of it like taking detailed notes on a plant to see how it grows and changes over time, without trying to make it grow differently.

Researchers will follow a group of boys with DMD, aged between 4 and 9 years old, for a period ranging from 6 months to up to 3 years. During this time, they will use a set of standard tests and evaluations to see how the disease develops. The main reason for doing this is to gather important information about the typical course of DMD in this age group. This baseline data is incredibly valuable because it will help with future studies, especially those investigating potential new treatments like gene therapy. By knowing the natural progression, scientists can better tell if a new treatment is actually making a difference.

Another important aim of this study is to identify specific signs or measurements – whether they come from physical exams, scans, or blood tests – that might predict how the disease will progress. They also want to figure out the best ways to measure changes in DMD during future clinical trials. All this effort is about laying the groundwork to develop more effective treatments and improve the lives of those affected by Duchenne Muscular Dystrophy.

Key takeaways

The study observes Duchenne Muscular Dystrophy (DMD) in boys aged 4-9.
It aims to understand how DMD naturally progresses over time.
No new treatments are given; focus is on observation and assessment.
Information gathered will help develop future treatments for DMD.
Participation involves regular clinic visits and various tests.
You can withdraw from the study at any point without affecting care.

Who may be eligible?

This study is looking for boys with Duchenne Muscular Dystrophy (DMD) who are between 4 and 9 years old. They need to have a confirmed diagnosis of DMD through a genetic test. Participants should also be able to perform certain physical tasks, like walking a specific distance in a 6-minute test or showing particular scores on movement assessments. It's important that they are already taking corticosteroids or are just starting them as part of their usual care.

There are also some things that would mean a boy couldn't join the study. For example, if he has certain heart problems, needs help with breathing (like a breathing machine day or night), or has other health issues that might affect how DMD progresses or how the study tests can be done. Boys who cannot cooperate with muscle tests or have metal implants that would interfere with MRI scans also wouldn't be able to participate. Finally, families must be willing and able to follow all the study's instructions and provide their agreement for their child to take part.

Quick self-check

Is your child a boy?
Is he between 4 and 9 years old?
Does he have a confirmed diagnosis of DMD through a genetic test?
Is he able to walk a certain distance or perform specific movement tasks?
Is he currently taking, or just starting, corticosteroid medication?
Does he not have severe heart or breathing issues that would prevent study participation?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If your child takes part in this study, they will have regular visits to the clinic over a period of 6 months to 3 years. These visits will involve various assessments to track their progress. This might include physical examinations, muscle tests, and possibly scans like an MRI, if appropriate. They will be asked to do activities like walking tests and other movements to see how their muscles are performing. There are no new medications given as part of this study; participants will continue on their usual corticosteroid therapy. The main aspect of participation is careful observation and assessment. The total duration for each child will vary, but it could be up to 36 months.

Potential risks and benefits

Taking part in this study won't offer a new treatment for your child, but the information gathered could greatly help future research into Duchenne Muscular Dystrophy, potentially leading to better treatments for others. Your child will receive regular health checks and close monitoring by medical professionals. Potential risks are generally low, mainly related to the inconvenience of clinic visits and the possibility of discomfort from standard physical assessments or scans like MRI. You are free to withdraw your child from the study at any time, for any reason, without it affecting their medical care.

Locations (15)

Centre Hospitalier Universitaire Brugmann
Brussels, Belgium· Recruiting
UZ Leuven
Leuven, Belgium· Recruiting
CHR Hôpital de la Citadelle
Liège, Belgium· Not yet recruiting
University Hospital of Bordeaux
Bordeaux, France· Recruiting
Brest University Hospital Centre
Brest, France· Recruiting
Hopital Femme Mere Enfant
Bron, France· Recruiting
CHU Lille
Lille, France· Recruiting
Hopital la Timone Enfants
Marseille, France· Recruiting
Centre Hospitalier Universitaire - Hôpital Gui de Chauliac
Montpellier, France· Not yet recruiting
Hôpital Armand Trousseau
Paris, France· Recruiting
Hôpital Hautepierre
Strasbourg, France· Recruiting
Hospital Sant Joan de Deu Esplugues de Llobregat
Esplugues de Llobregat, Spain· Recruiting

+3 more sites — see the official record for the full list.

Common questions

What is the main goal of this study?

The main goal is to understand how Duchenne Muscular Dystrophy naturally progresses in boys aged 4 to 9, without testing a new treatment.

Will my child receive any new medication in this study?

No, this study does not involve new medications. Your child will continue with their usual corticosteroid treatment.

How long will my child be in the study?

Participation can last anywhere from 6 months to up to 3 years, depending on the individual.

What kind of tests will my child have?

They will have physical exams, muscle strength tests, walking tests, and possibly MRI scans to monitor their condition.

Can we stop participating if we change our minds?

Yes, you can withdraw your child from the study at any time, and it won't affect their regular medical care.

How to find out more

Francesco MUNTONI, Pr

c.griffith@ucl.ac.uk +44 02079052602 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.